JP2023522701A - Ｍｓｈ３調節のためのオリゴヌクレオチド - Google Patents

Ｍｓｈ３調節のためのオリゴヌクレオチド Download PDF

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JP2023522701A
JP2023522701A JP2022563451A JP2022563451A JP2023522701A JP 2023522701 A JP2023522701 A JP 2023522701A JP 2022563451 A JP2022563451 A JP 2022563451A JP 2022563451 A JP2022563451 A JP 2022563451A JP 2023522701 A JP2023522701 A JP 2023522701A
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nucleotides
dsrna
antisense strand
strand
sense strand
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JP2023522701A5 (https=
JPWO2021216556A5 (https=
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コボロバ，アナスタシア
ファーガソン，シャンタル
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University of Massachusetts Amherst
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
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  • Animal Behavior & Ethology (AREA)
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JP2022563451A 2020-04-20 2021-04-20 Ｍｓｈ３調節のためのオリゴヌクレオチド Pending JP2023522701A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063012603P 2020-04-20 2020-04-20
US63/012,603 2020-04-20
PCT/US2021/028166 WO2021216556A2 (en) 2020-04-20 2021-04-20 Oligonucleotides for msh3 modulation

Publications (3)

Publication Number Publication Date
JP2023522701A true JP2023522701A (ja) 2023-05-31
JP2023522701A5 JP2023522701A5 (https=) 2024-05-02
JPWO2021216556A5 JPWO2021216556A5 (https=) 2024-05-02

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JP2022563451A Pending JP2023522701A (ja) 2020-04-20 2021-04-20 Ｍｓｈ３調節のためのオリゴヌクレオチド

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Country Link
US (1) US20210355491A1 (https=)
EP (1) EP4138860A4 (https=)
JP (1) JP2023522701A (https=)
AU (1) AU2021260581A1 (https=)
CA (1) CA3176204A1 (https=)
WO (1) WO2021216556A2 (https=)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3277811B1 (en) 2015-04-03 2020-12-23 University of Massachusetts Fully stabilized asymmetric sirna
US10633653B2 (en) 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
JP7627042B2 (ja) 2018-08-23 2025-02-05 ユニバーシティー オブ マサチューセッツ Ｏ－メチルリッチ完全安定化オリゴヌクレオチド
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
MX2022001710A (es) 2019-08-09 2022-05-10 Univ Massachusetts Oligonucleótidos modificados químicamente dirigidos a los snp.
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
WO2021226549A1 (en) * 2020-05-08 2021-11-11 Triplet Therapeutics, Inc. Methods for the treatment of nucleotide repeat expansion disorders associated with msh3 activity
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
AR122534A1 (es) 2020-06-03 2022-09-21 Triplet Therapeutics Inc Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3
EP4164656A4 (en) * 2020-06-11 2025-10-22 Ionis Pharmaceuticals Inc COMPOUNDS AND METHODS FOR REDUCING MSH3 EXPRESSION
WO2022212231A2 (en) 2021-03-29 2022-10-06 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
WO2023076450A2 (en) * 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
US20230313198A1 (en) * 2021-12-17 2023-10-05 University Of Massachusetts Oligonucleotides for mlh3 modulation
US20240035027A1 (en) * 2022-05-04 2024-02-01 University Of Massachusetts Oligonucleotides for pms2 modulation
AU2023272008A1 (en) * 2022-05-16 2024-12-12 Atalanta Therapeutics, Inc. Compositions and methods for treatment of microsatellite dna expansion disorders
WO2024064954A2 (en) * 2022-09-23 2024-03-28 The Children's Hospital Of Philadelphia Compositions and methods for treating huntington's disease and related disorders
WO2025078602A1 (en) * 2023-10-11 2025-04-17 Uniqure Biopharma B.V. Nucleic acid regulation of msh3 in repeat expansion disorders

Citations (3)

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Publication number Priority date Publication date Assignee Title
US20150307876A1 (en) * 2012-02-10 2015-10-29 Regents Of The University Of Minnesota Dna assimilation
US20170183655A1 (en) * 2014-05-07 2017-06-29 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Compositions and uses for treatment thereof
JP2019503394A (ja) * 2016-01-31 2019-02-07 ユニバーシティ・オブ・マサチューセッツＵｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍａｓｓａｃｈｕｓｅｔｔｓ 分岐オリゴヌクレオチド

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AUPO974597A0 (en) * 1997-10-10 1997-11-06 Rhone-Poulenc Agro Methods for obtaining plant varieties
US7361468B2 (en) * 2004-07-02 2008-04-22 Affymetrix, Inc. Methods for genotyping polymorphisms in humans
TN2017000354A1 (en) * 2015-02-10 2019-01-16 Genzyme Corp VARIANT RNAi
SMT202000454T1 (it) * 2015-04-03 2020-11-10 Univ Massachusetts Cmposti oligonucleotidici per bersagliare l'mrn della huntingtina
US20170009304A1 (en) * 2015-07-07 2017-01-12 Splicingcodes.Com Method and kit for detecting fusion transcripts
JP7714162B6 (ja) * 2018-12-03 2025-09-08 タケダ ファーマシューティカルズ ユーエスエー インコーポレイテッド Ｍｓｈ３活性に関連するトリヌクレオチドリピート伸長障害の処置のための方法

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150307876A1 (en) * 2012-02-10 2015-10-29 Regents Of The University Of Minnesota Dna assimilation
US20170183655A1 (en) * 2014-05-07 2017-06-29 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Compositions and uses for treatment thereof
JP2019503394A (ja) * 2016-01-31 2019-02-07 ユニバーシティ・オブ・マサチューセッツＵｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍａｓｓａｃｈｕｓｅｔｔｓ 分岐オリゴヌクレオチド

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
ANNE-MARIE M. GANNON ET AL., NUCLEIC ACIDS RESEARCH, vol. 40, no. 20, JPN6025012917, 2012, pages 10324 - 10333, ISSN: 0005748786 *
NIMRAT CHATTERJEE ET AL., DNA REPAIR, vol. 42, JPN6025012916, 2016, pages 26 - 32, ISSN: 0005748787 *

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Publication number Publication date
EP4138860A2 (en) 2023-03-01
WO2021216556A2 (en) 2021-10-28
EP4138860A4 (en) 2024-08-07
US20210355491A1 (en) 2021-11-18
CA3176204A1 (en) 2021-10-28
AU2021260581A1 (en) 2022-11-24
WO2021216556A3 (en) 2022-03-03

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